Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain

Experiment: Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain

PI: Jason D Heaney, PhD

Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing. Editing detected and quantified in brain by generation of tdTomato fluorescent protein signal from Ai9 reporter mice

Delivery Assays:

- Identification of of GFP-positive nuclei (green)

Editing Assay:

- Qualitative assesment of tdTomato-negative cells throughout the entire brain
- Quantitative assessment of tdTomato-positive cells per DAPI-stained nucleus in a specific brain region
- Qualitative assesment of prevalence of tdTomato-positvie cells throughout the entire brain

Organ System Overview

Arm1a: BI28 Neuron Specific Promoter, Experimental Animals, Males

Arm1a: BI28 Neuron Specific Promoter, Experimental Animals, Females

Arm 1b: BI28 Neuron Specific Promoter, Positive Delivery Control Animals, Males

Arm 1b: BI28 Neuron Specific Promoter, Positive Delivery Control Animals, Females

Arm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Males

Arm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Females

Arm 2b: BI28 Astrocyte Specific Promoter, Positive Delivery Control Animals, Males

Arm 2b: BI28 Astrocyte Specific Promoter, Positive Delivery Control Animals, Females

Arm 3: Negative Control Saline, Males

Legend:

	Delivery Efficiency
	Editing Efficiency
	Target Tissue
	Not Available

Analyze Data Sets Available for this Experiment
Nervous
	Brain (neuron) (TARGET)
	Cerebral cortex (astrocyte) (TARGET)
	Dorsal plus ventral thalamus (astrocyte) (TARGET)
	Brain (astrocyte) (TARGET)
	Brain (unspecified)

Contact SCGE

Experiment: Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain